Understanding a rare disease is the first step in treating it. How can you combat the underlying mechanisms of a disorder if you do not know what they are? A…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Cannabidiol (CBD) is a chemical compound found in cannabis and derived from hemp. Unlike THC, CBD is not psychoactive. It is lauded for its ability to treat a wide variety…
According to a recent article, researchers were able to produce the first images of an enzyme that may figure out the unknown cause of a rare group of hereditary neurodegenerative…
According to a news release from immuno-oncology company IMV Inc., the first patient has been dosed in the Phase 2b VITALIZE clinical trial. During the trial, researchers are evaluating a…
Over the last two years, the COVID-19 pandemic has dominated the headlines. In 2021, vaccines became available to prevent severe COVID-19 infection. However, this has also brought up numerous questions…
Moebius Syndrome Awareness Day is January 24th! This event was created in 2011 by the Many Faces of Moebius Syndrome (MFOMS). It is now celebrated across the world, as the…
In late December 2021, biopharmaceutical company Deciphera Pharmaceuticals, Inc. ("Deciphera") shared that its therapy QINLOCK (ripretinib) was approved in the United Kingdom. The treatment is designed for adults with advanced…
BeyondSpring Pharmaceuticals has issued a press release announcing that the FDA presented the company with a complete response letter pertaining to its drug plinabulin and granulocyte colony-stimulating factor (G-CSF). The…
The Klassen family is no stranger to the rare disease community. According to the Denver Post, Greg Klassen was first diagnosed with spinocerebellar ataxia type 3 (SCA3) nearly ten years…
This week the Guardian reported that a pig’s heart, which was genetically modified, had been transplanted into a patient by doctors who were trying to save his life. The patient was…
According to a recent article, Castle Creek Inc. has acquired Novavita Thera Inc. in an effort to expand its research and development efforts. Castle Creek Inc. Castle Creek Biosciences Inc.…
A glioblastoma diagnosis can be devastating. Currently, the 5-year survival rate is under 10%. This highly malignant cancer, which forms in the brain and spinal cord, is also somewhat difficult…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Grant and award programs can be crucial in spurring therapeutic discovery and development. According to News Medical, this is what the Follicular Lymphoma Foundation (FLF) aims to do with its…
Recently, Chain Drug Review reported that the FDA had approved a generic form of colchicine pills (normally prescribed as Mitigare, Colcrys, or Gloperba) for the treatment of familial Mediterranean fever (FMF).…
According to a recent article, the drug dovitinib has been submitted to the FDA for pre-marketing approval as a third-line therapy for patients diagnosed with renal cell carcinoma (RCC). Renal…
According to the Cancer Network, a CAR T-cell therapy called C-CAR039 recently earned both Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration.…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
It's January -- do you know what that means? That's right - it's National Glaucoma Awareness Month! According to the Richmond Register, glaucoma diagnoses are rising on a national level.…
On January 12, 2022, posts shared on Facebook and Instagram announced the passing of 15-year-old Adalia Rose Williams. A Youtube and Facebook star, Adalia had over 16 million followers collectively.…
Crohn’s disease causes inflammation throughout the digestive tract. But what factors influence the severity of this inflammation? Medical XPress describes how a study out of Case Western Reserve University discovered…
There are many terms that were once widely used which held offensive origins and meanings towards certain groups. Now, in a more socially conscious society, we are rightfully abandoning these…
According to a recent article from MedPage Today, the results from a small prospective study show that sequential radiotherapy (RT) is successful in delaying systemic therapy for oligometastatic renal cell…
In the past, there have been little to no concrete studies evaluating the relationship between hereditary angioedema (HAE) and cancer risk. However, researchers questioned whether some sort of relationship could…
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